148 articles
Istradefylline
Istradefylline (NOURIAST, Nourianz; KW-6002) is a first-in-class adenosine A2A receptor antagonist taken once daily with levodopa for OFF episodes in Parkinson's disease; approved in Japan 2013 and by the FDA in 2019.
Lacosamide
Lacosamide (Vimpat) is a third-generation antiseizure medication, a functionalized amino acid that selectively enhances slow inactivation of voltage-gated sodium channels; approved 2008 for focal seizures and primary generalized tonic-clonic seizures.
Lamotrigine
Lamotrigine (Lamictal), a phenyltriazine anticonvulsant approved in the US in 1994, treats focal and generalized seizures and maintains bipolar I disorder; sodium-channel blocker with boxed warning for serious rash and major valproate and contraceptive interactions.
Lanabecestat
Lanabecestat (AZD3293, LY3314814) is an oral BACE1 inhibitor developed by AstraZeneca and Eli Lilly for early Alzheimer disease; its AMARANTH and DAYBREAK-ALZ trials failed and the program was discontinued in June 2018.
Laquinimod
Laquinimod (ABR-215062, Nerventra) is an oral quinoline-3-carboxamide immunomodulator activating the aryl hydrocarbon receptor, trialed for multiple sclerosis (EMA-refused 2014), Huntington's disease, and now inflammatory eye disorders.
Lasmiditan
Lasmiditan (Reyvow, formerly COL-144/LY573144) is a selective 5-HT1F receptor agonist, the first ditan, FDA-approved 11 October 2019 for acute migraine in adults; non-vasoconstricting, 50-200 mg, one dose per 24 h, 8-hour driving restriction, Schedule V.
Latrepirdine
Latrepirdine (Dimebon, dimebolin), a Soviet 1983 antihistamine repurposed for Alzheimer's and Huntington disease; strong phase II results led to a Pfizer deal, but phase III trials failed.
Lecozotan
Lecozotan (WAY-161333, SRA-333) is a silent 5-HT1A receptor antagonist developed by Wyeth for mild-to-moderate Alzheimer's disease; it potentiated hippocampal glutamate and acetylcholine release, passed phase I trials, but development was suspended after phase 2.
Levacetylleucine
Levacetylleucine, also known as N-acetyl-L-leucine and marketed as Aqneursa, is an oral drug approved in 2024 for neurological symptoms of Niemann-Pick disease type C and in 2026 for ataxia in ataxia-telangiectasia.
Licarbazepine
Licarbazepine (10-hydroxycarbazepine, MHD) is the chiral active metabolite of the antiepileptics oxcarbazepine and eslicarbazepine acetate; S-enantiomer (eslicarbazepine) predominates, half-life about 8-10 hours, sodium-channel blocker.
Low-dose naltrexone
Low-dose naltrexone (LDN) is the off-label use of the opioid antagonist naltrexone at roughly 0.5 to 4.5 mg daily, investigated mainly for chronic pain, inflammation, and long COVID.
Masupirdine
Masupirdine (SUVN-502) is Suven Life Sciences' investigational 5-HT6 receptor antagonist, failed a Phase 2 Alzheimer's cognition trial, and is now in a 375-patient Phase 3 trial for agitation in Alzheimer's dementia.
Memantine
Memantine (Namenda, Ebixa, Axura) is a moderate-affinity uncompetitive NMDA receptor antagonist, first FDA-approved in 2003 for moderate-to-severe Alzheimer's disease; small symptomatic benefit, no benefit in mild AD, generic since 2015.
Mephenytoin
Mephenytoin (Mesantoin), 3-methyl-5-ethyl-5-phenylhydantoin, a hydantoin anticonvulsant marketed since 1945 for partial and secondarily generalized seizures; metabolized to nirvanol, limited by idiosyncratic toxicity, and used as a CYP2C19 probe drug.
Mephobarbital
Mephobarbital (methylphenobarbital, Mebaral, Prominal) is an N-methyl barbiturate anticonvulsant and sedative introduced in 1932, metabolized to phenobarbital, Schedule IV, now discontinued in the US.
Metharbital
Metharbital (Gemonil, endiemal) is a long-acting barbiturate anticonvulsant, 5,5-diethyl-1-methylbarbituric acid, patented 1905 by Emil Fischer of Merck, marketed by Abbott from 1952, discontinued 1990, demethylated to barbital, used mainly for infantile myoclonic spasms.
Methsuximide
Methsuximide (Celontin), an anticonvulsant succinimide chemically known as N,2-dimethyl-2-phenylsuccinimide, treats absence seizures refractory to other drugs; FDA-approved 1957 by Parke-Davis, active via N-desmethylmethsuximide metabolite.
Methylphenidate
Methylphenidate (Ritalin, Concerta) is a racemic CNS stimulant and dopamine/noradrenaline reuptake inhibitor approved for ADHD ages 6+ and narcolepsy; SMD −0.78 efficacy, ~24M US prescriptions, Schedule II, WHO essential medicine 2023.
Modafinil
Modafinil (Provigil) is a Schedule IV wakefulness-promoting drug approved in 1998 for narcolepsy sleepiness, also used for OSA and shift work disorder in the US but restricted to narcolepsy in the EU.
Motivation-enhancing drug
Motivation-enhancing drug: agents aimed at apathy and motivation deficits, chiefly methylphenidate and dopaminergic drugs targeting incentive salience; no treatment is yet licensed for apathy.
Mouse nerve growth factor
Mouse nerve growth factor (鼠神经生长因子, mNGF) is a 13.5 kDa NGF protein extracted from mouse submandibular glands, marketed in China as a category I injectable biologic for optic nerve injury and other neurological conditions.
Naratriptan
Naratriptan (Amerge) is an oral 5-HT1B/1D receptor agonist triptan for acute migraine in adults, US-approved in 1998; 1 and 2.5 mg doses, 5-6 hour half-life, tolerability similar to placebo.
Neramexane
Neramexane (MRZ 2/579), a Merz Pharmaceuticals amino-alkyl-cyclohexane drug, is an uncompetitive NMDA receptor channel blocker and alpha9alpha10 nicotinic antagonist trialed for tinnitus, Alzheimer's disease, pain, and addiction, without approval.
Nerinetide
Nerinetide (NA-1, Tat-NR2B9c), a 20-amino-acid PSD-95 inhibitor neuroprotectant peptide for acute ischemic stroke; neutral in phase 3 ESCAPE-NA1, degraded by alteplase-generated plasmin, with benefit signals in alteplase-free subgroups.
NNZ-2591
NNZ-2591 (ercanetide), also called cyclo-L-glycyl-L-2-allylproline, is Neuren Pharmaceuticals' oral IGF-1 metabolite analog in Phase 3 (Koala trial) for Phelan-McDermid syndrome and in development for Pitt Hopkins, Angelman, HIE, and SYNGAP1-related disorder.
Nootropic
Nootropics, also called smart drugs or cognition enhancers, are substances meant to improve memory, attention, and learning; the term was coined in 1972, and evidence of benefit in healthy adults is weak.
NTI-164
NTI164 (NTI-164) is Neurotech International's CBDA-dominant full-spectrum low-THC oral cannabinoid therapy for pediatric neuroinflammatory disorders, tested in ASD (Harmony Phase II/III), Rett syndrome, PANDAS/PANS, and cerebral palsy, now in Phase 3 Beyond Harmony.
Olesoxime
Olesoxime (TRO19622), a cholesterol-oxime mitochondrial neuroprotectant from Trophos later owned by Roche, was trialed in SMA and ALS but showed no significant benefit and development ended in 2018.
Omaveloxolone
Omaveloxolone (Skyclarys, RTA 408) is a semisynthetic oleanane triterpenoid Nrf2 activator, the first drug approved for Friedreich's ataxia (FDA 2023, EU 2024), dosed 150 mg once daily.
Opicapone
Opicapone (Ongentys) is a third-generation, once-daily, peripheral reversible COMT inhibitor used with levodopa/carbidopa for OFF episodes in Parkinson's disease; approved in the EU (2016), US (2020), and Japan (2020).